(322) Highly-effective modulator therapies: Impact on the well-being of people living with cystic fibrosis and implications for the CF care model

Background: A number of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies have recently been approved for people living with cystic fibrosis (PwCF). PwCF using these highly-effective modulator therapies (HEMT) have shown significant improvements in quality of life and clinical disease markers, including lung function; and research suggests that such therapies can slow or prevent the progression of symptoms among PwCF. As a result of such significant clinical changes, there is a pressing need to understand the related impact on quality of life and to identify potential implications for the CF care model of the future.

Methods: Study design: We will conduct a needs assessment to explore the impact of HEMT on well-being and quality of life among PwCF and emerging concerns and issues that matter most to PwCF using HEMT. We will deploy an electronic survey that assesses health-related needs and assets among PwCF, changes since starting HEMT (if applicable), and guidance for future research and care model changes. Measurement domains and questions will be developed in partnership with CF Foundation staff and leadership, clinician-researchers, and patient and family partners. We will use a mixed-methods framework to analyze categorical and narrative survey data. Patient selection criteria: Data will be collected using an electronic survey distributed through established CF Foundation communication channels, including social media (e.g., Twitter and Facebook posts), targeted emails (e.g., Community Voice), general e-mail mentions (e.g., CF Foundation newsletters), and care team communications. Adults (ages 18+) with CF and caregivers of children with CF will be eligible to participate. Primary and secondary endpoints: Survey domains will assess physical well-being, emotional and psychological well-being, economic well-being, social well-being, treatment burden, and perceptions of the CF care delivery model. We will compare responses between individuals who have used HEMT and those who have not used HEMT.

Results: Interim results are not available at this time. Data collection will be finalized on July 30, 2022.

Conclusions: By conducting a mixed-methods evaluation of PwCF experiences and perceptions associated with the use of HEMT, we will lay the groundwork for establishing future research priorities, policy efforts, and communications. This work will be co-designed by PwCF and their caregivers, clinicians, and researchers to identify those areas that matter most and that have the greatest potential impact for improving health-related quality of life for PwCF using HEMT.

Acknowledgements: This work was supported by the Cystic Fibrosis Foundation [grant number NELSON20QI0].

References: